The False Hope Of Colorado's 'Right To Try' Investigational Drug Law

On Saturday, Governor John Hickenlooper signed into law Colorado House Bill 14-1281, The Right to Try Act. The bill was co-sponsored by a retired physician and supported by others who wish to improve access to unapproved, investigational drugs for terminally-ill patients.

While supporters claim that the bureaucracy surrounding "compassionate use" of drugs undergoing clinical trials is too burdensome for patients at imminent risk of death, the new law really does nothing to increase the odds that a patient will have access to an experimental therapy.

Section 25-45-104 of the bill states,

"A manufacturer of an investigational drug, biological product, or device may make available the manufacturer's investigational drug, biological product, or device to eligible patients pursuant to this article. This article does not require that a manufacturer make available an investigational drug, biological product, or device to an eligible patient." (full PDF here)

The second sentence of the Colorado bill also grants what federal policy already does: Companies trying to get a drug to market as quickly as possible are under no legal obligation to provide a requestor with the drug or device.

I have covered other compassionate use cases earlier this year and spoken with family members seeking expanded access to such drugs. Mikaela Knapp, a 25-year-old woman with metastatic renal cell carcinoma, passed away a month after her husband and friends mounted a tremendous campaign to obtain access to a type of drug currently under development by three separate companies.

Although none of the companies made formal statements defending their decisions not to offer the drug to Mikaela, the most likely reason was that her disease was far too advanced for any therapy to make a difference. One issue that's glossed over in this whole discussion is that an incompletely tested drug given to an acutely, terminally-ill patient might actually hasten their death and/or increase their suffering.

"Flat-out cruel"

I submit that this seemingly well-meaning but meaningless Colorado act does nothing but create a sense of false hope for similar families. The act does nothing more than assuage the concerns of lawmakers that they haven't done enough to help their constituents. Instead, they've done a disservice.

Today at MSNBC.com, NYU bioethicist Arthur Caplan pulled no punches in writing that passing such bills as Colorado's was "flat-out cruel." He cited Colorado State Senator Irene Aguilar, a physician, as saying the bill was inspired by the movie, The Dallas Buyer's Club, a story set 30 years ago for patients with AIDS to gain access to HIV drugs illegal by importation through Mexico. However, the movie was set before the FDA established compassionate use parameters.

States cannot trump federal drug regulatory authority, and we already have the federal equivalent of #RightToTry (as it's being hashtagged on Twitter). The compassionate use provision, officially known as "expanded access to investigational drugs outside of a clinical trial," already allows for companies to provide experimental drugs, if available, on a case-by-case basis, and with FDA's approval. Even with the federal provision, companies are not compelled to provide the drug.

In addition to this allowance, the FDA has also provided other mechanisms to get drugs approved and available to patients as quickly as possible via fast-track or "breakthrough therapy" designation. But we bear a responsibility as a society to have a reasonable picture of a drug's safety or effectiveness before offering it even for a last-ditch effort.